I blame alot of the on Hemispherix. I'd like to know why they did not do the larger clinical trial that the FDA asked them to do a couple years ago when Ampligen was denied. And why did the FDA accept their application for review knowing that this wasn't done. I can't stand the wasted years on this - we are back to where we were before - lots of time, lots of energy wasted!!

In its Complete Response Letter ("CRL"), the FDA set forth the reasons for this action and provided recommendations to address certain of the outstanding issues. The Agency stated that the submitted data do not provide substantial evidence of efficacy of Ampligen® for the treatment of CFS and that the data do not provide sufficient information to determine whether the product is safe for use in CFS due to the limited size of the safety database and multiple discrepancies within the submitted data.

In the two pivotal clinical studies that form the basis of approval for Ampligen®, Hemispherx believes that the primary efficacy endpoints were met and that they showed a statistically significant improvement (i.e., with a p-value of 0.05 or less). The FDA and Hemispherx do agree that in clinical study AMP-502, the primary endpoint was met (p=0.02). In clinical study AMP-516, the FDA's analysis resulted in a p-value of 0.10, while Hemispherx's calculation resulted in a p-value of <0.05, and yet both analyses indicate that those patients on Ampligen® improved over those on placebo. With regard to safety, Hemispherx has provided data from the 845 subjects who have received Ampligen®, including 589 subjects suffering from severe CFS and over 200 CFS patients who have received Ampligen® for at least one year or longer. The Company believes that these data are sufficient to determine the safety profile of Ampligen®. At the December 20, 2012 FDA Advisory Committee meeting, 8 of the 13 Advisory Committee members voted yes on the question of "Is the safety profile of Ampligen® adequate for approval for the treatment of CFS?"

Hemispherx plans to request an end-of-review conference with the FDA as a precursor to submitting a formal appeal to the Office of New Drugs in the FDA's Center for Drug Evaluation and Research regarding the Agency's decision. The purpose of the conference is to review all of the issues raised in the Agency's CRL as well as to discuss the corroborating data and experiences of clinicians and patients who have seen the benefits of Ampligen® therapy.

Hemispherx has become aware that a prominent CFS advocate and long-time CFS sufferer, who has been on Ampligen® since 1999 through a treatment IND, began a hunger strike on January 30, 2013 to seek FDA approval of Ampligen®. Hemispherx understands the frustration that there is still no FDA-approved treatment for CFS and the concern that patients may lose access to Ampligen® therapy. Out of concern for the health of the CFS community, Hemispherx has asked any hunger strikes be discontinued and that patients join in a collaborative effort between the FDA, Hemispherx, CFS clinicians and patient advocates to find a solution to this significant unmet medical need.

In the past, the FDA has shown great willingness to work with stakeholders to find solutions for serious and life-threatening illnesses. Dr. Margaret Hamburg, Commissioner of the FDA has previously stated that, "FDA has an important role to play in shaping the future of medical breakthroughs by bringing stakeholders together to identify and overcome challenges." Hemispherx hopes that the FDA will view the Ampligen® end-of-review conference as an opportunity to involve patient advocacy, clinicians and researchers in a concentrated effort to do something for these patients over the near-term, including further evaluation of how new legislation, such as the recently enacted "FDASIA" statute, may have a role in finding a solution. The views of one internationally recognized researcher/clinician, Dr. Nancy Klimas, who has over 20 years' experience evaluating and treating CFS patients, can be found at http://www.sciencedaily.com/releases/2013/01/130124183448.htm

DISCLOSURE NOTICE: The information in this press release and the article referenced therein includes certain "forward-looking" statements (explained below), including statements about the remaining steps, including the aforementioned end-of-review conference and appeals process, which the FDA may require and Hemispherx may take in further seeking FDA approval of the Ampligen® NDA for the treatment of Chronic Fatigue Syndrome. The final results of these and other ongoing activities could vary materially from Hemispherx's expectations and could adversely affect the chances for approval of the Ampligen® NDA. Any failure to satisfy the FDA's requirements could significantly delay, or preclude outright, approval of the Ampligen® NDA.

About Hemispherx Biopharma

Hemispherx Biopharma, Inc. is an advanced specialty pharmaceutical company engaged in the manufacture and clinical development of new drug entities for treatment of seriously debilitating disorders. Hemispherx's flagship products include Alferon N Injection® (FDA approved for a category of sexually transmitted diseases) and the experimental therapeutics Ampligen® and Alferon® LDO. Because both Ampligen® and Alferon® LDO are experimental in nature, they are not designated safe and effective by a regulatory authority for general use and are legally available only through clinical trials with the referenced disorders. Ampligen® is an experimental RNA nucleic acid being developed for globally important debilitating diseases and disorders of the immune system including Chronic Fatigue Syndrome. Hemispherx's platform technology includes components for potential treatment of various severely debilitating and life threatening diseases. Hemispherx has patents comprising its core intellectual property estate and a fully commercialized product (Alferon N Injection®). The Company wholly owns and exclusively operates a GMP certified manufacturing facility in the United States for commercial products. For more information please visit www.hemispherx.net.

Forward-Looking Statements

To the extent that statements in this press release are not strictly historical, all such statements are forward-looking, and are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Words such as "believes," "plans," "anticipates," and similar expressions are intended to identify forward-looking statements. These statements are based on the company's current beliefs and expectations and represent the Company's judgment as of the date of this release. The inclusion of forward-looking statements should not be regarded as a representation by Hemispherx that any of its plans will be achieved, including its intent to pursue the end-of-review conference and appeals process. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond Hemispherx's control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. Examples of such risks and uncertainties include those set forth in the Disclosure Notice, above, as well as the risks described in Hemispherx's filings with the Securities and Exchange Commission, including the most recent reports on Forms 10-K, 10-Q and 8-K and Hemispherx's beliefs that the Ampligen® NDA may be covered by the new provisions of the FDASIA statute, which are subject to FDA interpretation and implementation, or that such provisions, if applicable, will be helpful with regard to obtaining FDA approval of the Ampligen® NDA. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Hemispherx undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise revise or update this release to reflect events or circumstances after the date hereof.

"Hemispherx plans to request an end-of-review conference with the FDA as a precursor to submitting a formal appeal to the Office of New Drugs in the FDA's Center for Drug Evaluation and Research regarding the Agency's decision. The purpose of the conference is to review all of the issues raised in the Agency's CRL as well as to discuss the corroborating data and experiences of clinicians and patients who have seen the benefits of Ampligen® therapy.

[...] Hemispherx has asked [...] that patients join in a collaborative effort between the FDA, Hemispherx, CFS clinicians and patient advocates to find a solution to this significant unmet medical need.

[...] Hemispherx hopes that the FDA will view the Ampligen® end-of-review conference as an opportunity to involve patient advocacy, clinicians and researchers in a concentrated effort to do something for these patients over the near-term, including further evaluation of how new legislation, such as the recently enacted "FDASIA" statute, may have a role in finding a solution. "​

And there's that two-day webinar that was yesterday and today about how the FDA can fast-track treatments that sounded remarkably like Ampligen.

I don't think we're back to square on this - and we've come a very long way in a year.

Without approval could doctors still prescribe off-label? I'm assuming this is unlikely as the drug is so expensive (can't help thinking that's at the root of the refusal; neither insurers nor the vastly cut budget of Medicaid want to pay).

As for why Hemiwhatsits didn't do the bigger study, I was under the impression it was because they were skint.
The bigger study set up in Canada (as far as I remember) was only for patients who could pay the astronomical amount required. Hardly surprising it didn't get off the ground.

I'm not surprised at this outcome. I am at the point of believing this is all about egos and money and has nothing to do with ME. All they want is not to be proved wrong and allowing Ampligen is admitting they were pillocks of the first order.

A protocol of tests that all patients suspected of having ME (and fibro for that matter) must be put through needs to be established first. When we all or nearly all test positive for an agreed set of viruses and reativation antibodies along with results on high res SPECT and other scans there may be hope.

I blame alot of the on Hemispherix. I'd like to know why they did not do the larger clinical trial that the FDA asked them to do a couple years ago when Ampligen was denied. And why did the FDA accept their application for review knowing that this wasn't done. I can't stand the wasted years on this - we are back to where we were before - lots of time, lots of energy wasted!!

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I don't know the exact facts but in my eyes it's very easy to blame a company for not doing some huge phase III trials. Do you know how expensive these trials are? You pay about 10,000 to 20,000 dollar per patient. Now multiply by 1000 patients. Who can afford this? Thousands of promising drugs are stuck after Phase II because the companies cannot afford to do Phase III trials. It would be the same, if you only allowed Premium cars on the roads because they have better safety records of whatever. It should be the patients choice, what he or she buys. Drugs that finish Phase II are safe, otherwise they wouldn't have finished, so why can't we buy them? The Phase III requirement is one of the biggest innovation destroyers on earth. Government wastes billions of dollars on all kinds of things, so when it requires Phase III after successful completion of Phase II, it should also pay for it or let patients decide, if they buy a drug or not.

"My HungerStrike continues. I am asking the Secretary of Health to start NIH clinical trials with Ampligen in collaboration with the sponsor to document how to identify responders and why Ampligen works for many of us who are so ill."

By 'the sponsor' I think he means Hemispherx.

There's a Day 7 video update from him on there (before he heard the news).

Hemispherex raised $8 million from selling it's own shares when at their peak - due to speculation about approval. Not saying this money is now available to them for trials but thought I'd throw it in the mix. They have more money now than they did before and for what it's worth I think Hemispherex were always in need of 'putting their money where their mouths' were.

"My HungerStrike continues. I am asking the Secretary of Health to start NIH clinical trials with Ampligen in collaboration with the sponsor to document how to identify responders and why Ampligen works for many of us who are so ill."

By 'the sponsor' I think he means Hemispherx.

There's a Day 7 video update from him on there (before he heard the news).

I almost feel like joining Bob.. Im feeling down right now due to not even being able to get to a dr for the past 6 weeks and once again needing to cancel one of my medical appointments on Friday as I have no one to get me there. The Disablity Services are just not recognising that I cant just go to the specialists at the nearest little hospital for help and need to see my own specialists for the very specialist areas I need.

I knew this wouldnt be approved at this point of time. Maybe the company will end up completely broke before its ever approved. The American gov should help pay for more studies of this then.

Please find below a Letter to me from Hemispherx the Sponsor of AmpligenFrom:Tom Equelsposted to Robert Millerabout an hour ago.

Dear Bob,

Hemishpherx Biopharma has just announced the FDA denial of the Ampligen new drug application. We will ask, within the next week, for an “End of Review Conference” as a precursor to our formal appeal of this denial. We are asking the FDA, in the spirit of your Day 6 video statement, to join with Hemispherx, CFS patients and clinical experts in CFS to find a SOLUTION to this devastating disease and life threatening unmet medical need.
We believe a hunger strike is far too dangerous given your medical condition and, on that basis alone; we implore you to end your hunger strike. However, we also know that if we are to have a meaningful End of Review Conference, and a REAL FDA commitment to seek a prompt and meaningful solution providing immediate therapeutic access for this devastating disease, we will need your active participation as a patient advocate.
Respectfully,

According to Twitter, Bob's hunger strike is now directed at the NIH, I assume to fund trials.

I think in lieu of approval, a government funded Ampligen trial is definitely a sensible goal. Of course like anyone, I'm worried about Bob's condition at this stage. If I don't eat I can't stand up without passing out so I can imagine he's probably almost permanently lying down, save being on camera at this point.

I can understand why he's doing it, Ampligen isn't exactly going to be the solution for everyone and I don't doubt for a second there are problems with it. It's about applying the same standard that is applied to many other illnesses and I do think there is some amount of traction that comes from showing an issue has become unbearable enough to engender some level of self sacrifice.

I think Bob may well of struck a note here, that stonewall everybody's been hitting for years is tiresome. I am curious to see if his actions inspire subsequent ones.

According to Twitter, Bob's hunger strike is now directed at the NIH, I assume to fund trials.

I think in lieu of approval, a government funded Ampligen trial is definitely a sensible goal. Of course like anyone, I'm worried about Bob's condition at this stage. If I don't eat I can't stand up without passing out so I can imagine he's probably almost permanently lying down, save being on camera at this point.

I can understand why he's doing it, Ampligen isn't exactly going to be the solution for everyone and I don't doubt for a second there are problems with it. It's about applying the same standard that is applied to many other illnesses and I do think there is some amount of traction that comes from showing an issue has become unbearable enough to engender some level of self sacrifice.

I think Bob may well of struck a note here, that stonewall everybody's been hitting for years is tiresome. I am curious to see if his actions inspire subsequent ones.

Click to expand...

I can't see why the NIH of our cash-strapped, debt-ridden country will come up with the money to fund an Ampligen trial. We should implore Bob to begin eating.

I'm still shocked by the news and not thinking straight, but I don't believe we should let this issue die.

That's a very interesting piece, making it clear that we're not back where we started. There's some interesting stuff looking forward:

There will be several opportunities for the CFS community to inform and influence policy at FDA.​

This spring the FDA will sponsor a meeting of stakeholders across the patient, academic research and industry research landscapes to discuss ways to accelerate CFS drug discovery, development and approval.​

​

FDA has stated on several public occasions, including at the Dec. 20, 2012 advisory committee meeting, that CFS meets its criteria as a “serious and/or life-threatening” condition, making drug applicants eligible for various expedited review and approval provisions within the law. ​

The FDA is also conducting a series of meetings designed to better understand the benefit-risk assessment in conditions for which little is known or understood about effective treatment. ​

​

CFS was included in the initial list of conditions proposed by the agency and the CFIDS Association participated in a public hearing held Oct. 25, 2012 to support its inclusion in the final list and is actively engaged in the process that will shape how information about benefit-risk is collected from patient and assessed by the agency. ​